Intellia Therapeutics Highlights Its 3-Year Strategic Priorities And Anticipated 2024 Key Milestones; Ended 2023 In A Strong Financial Position With Approximately $1B In Cash; Cash Runway Extended Into Mid-2026

Author: Benzinga Newsdesk | January 04, 2024 05:02pm

• Core priorities through 2026 include completion of Phase 3 study and BLA submission for NTLA-2002, completion of patient enrollment in the pivotal study of NTLA-2001, clinical validation of modular in vivo gene insertion technology and advancement of earlier-stage pipeline and platform innovations
• On track to dose the first patient in the pivotal Phase 3 MAGNITUDE trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy in Q1 2024
• Expect to present results from the Phase 2 study of NTLA-2002 for the treatment of hereditary angioedema (HAE) and to initiate the Phase 3 study in 2024
• On track to dose the first patient in the Phase 1 study of NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency (AATD)-associated lung disease in 2024
• Ended 2023 in a strong financial position with approximately $1.0 billion in cash; cash runway extended into mid-2026
  
   

CAMBRIDGE, Mass., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced its strategic priorities through 2026 and key anticipated 2024 milestones that support the company's mission to transform the lives of patients and bring forth a new era in medicine.

"2023 was a momentous year for Intellia as our lead investigational therapy, NTLA-2001, advanced into the final stage of clinical development for the treatment of transthyretin amyloidosis with cardiomyopathy," said Intellia President and Chief Executive Officer John Leonard, M.D. "Looking ahead, we will continue to turn groundbreaking science into real-world medicines by capitalizing on our extensive experience and capabilities to edit disease-causing genes. We are rapidly expanding the potential to treat an even larger range of diseases, including those that originate outside of the liver, by deploying our novel delivery and editing technologies. With our comprehensive gene editing toolbox, we are well-positioned to harness the full potential of CRISPR-based medicines for patients."

Recent Pipeline Advancement and Corporate Updates

• NTLA-2001 for Transthyretin (ATTR) Amyloidosis: Initiated and actively enrolling the pivotal Phase 3 MAGNITUDE trial in adults with ATTR amyloidosis with cardiomyopathy (ATTR-CM).
• NTLA-2002 for Hereditary Angioedema (HAE): Completed enrollment and dosing in the Phase 2 portion of the Phase 1/2 study in adults with HAE.
• NTLA-3001 for Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease: Submitted a clinical trial application to initiate a first-in-human Phase 1 study of NTLA-3001 ahead of previous Q1 2024 expectation.
• Organizational Streamlining: Following an internal strategic review, Intellia will be streamlining company-wide operations to further focus resources on key strategic priorities and programs. These changes will result in a pause of select exploratory research-stage programs and a workforce reduction of approximately 15%.
• Cash Runway: Intellia ended the fourth quarter of 2023 with approximately $1.0 billion in cash, cash equivalents and marketable securities. The cash position is expected to fund operations into mid-2026.
• Board Update: In December, Intellia announced the departure of John Crowley from its board of directors following his appointment as Chief Executive Officer of the Biotechnology Innovation Organization (BIO), the life sciences industry's leading trade and advocacy association.

2024 – 2026 Strategic Priorities  

1. Complete patient enrollment for pivotal studies of NTLA-2001 and NTLA-2002, including planned biologics license application (BLA) submission for NTLA-2002 in 2026;
2. Launch clinical trials for next wave of in vivo and ex vivo programs, validating CRISPR-based in vivo targeted gene insertion and an allogeneic cell engineering solution designed to avoid NK cell-mediated rejection; and
3. Expand the range of diseases that can be targeted by deploying new editing and delivery innovations, including advancing gene editing in tissues outside the liver and DNA writing technology.
   
    

Anticipated 2024 Milestones

• NTLA-2001 for ATTR Amyloidosis:
  • Dose the first patient in MAGNITUDE trial of NTLA-2001 for the treatment of ATTR-CM in Q1 2024.
  • Continue to open new sites and enroll patients, globally, in the MAGNITUDE study.
  • Prepare for the Phase 3 study of NTLA-2001 for the treatment of ATTR amyloidosis with polyneuropathy (ATTRv-PN) in 2024.
  • Present updated clinical data from the ongoing Phase 1 study of NTLA-2001 in 2024.
• NTLA-2002 for HAE:
  • Initiate the global pivotal Phase 3 trial of NTLA-2002 in 2H 2024, subject to regulatory feedback.
  • Present updated data from Phase 1 and new data from Phase 2 portion in 2024.
• NTLA-3001 for AATD-Associated Lung Disease:
  • Dose first patient in Phase 1 study of NTLA-3001 in 2024.

Presentation at the 42nd Annual J.P. Morgan Healthcare Conference

John Leonard, M.D., President and Chief Executive Officer of Intellia, will present a company overview at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco on Tuesday, January 9, at 5:15 p.m. PT (8:15 p.m. ET). A live webcast will be available through the Events and Presentations page of the Investors & Media section on Intellia's website, www.intelliatx.com. A replay of the webcast will be available on Intellia's website for a limited time following the conference.

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